Screening in Oculopharyngeal Muscular Dystrophy

NCT ID: NCT02877784

Last Updated: 2020-03-27

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 8 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2016-10-31
• Study Completion Date: 2017-08-31

Brief Summary

Oculopharyngeal muscular dystrophy (OPMD) is a rare myopathic disease that results in progressive degeneration of the oral and pharyngeal muscular, resulting in severe dysphagia and dysarthria. OPMD is considered a rare disease; therefore, limited research is available on the natural progression of the disease or the utility of biomarkers to identify swallowing impairment. The aim of this study is:

1. To identify accurate, reliable and non-invasive clinical markers of swallowing impairment

2. To determine the discriminate ability of these markers to identify impairments in swallow safety and swallowing efficiency.

Detailed Description

Participants will be recruited from the Neurology clinic at the University of Florida. The single evaluation will occur in the PIs research laboratory at the University of Florida, Gainesville, Florida.

Conditions

Oculopharyngeal Muscular Dystrophy

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Screening

Participants enrolled will undergo testing of the swallowing mechanism

Videofluoroscopic swallowing study

Intervention Type: PROCEDURE

Videofluoroscopic swallowing study will be performed to measure the oropharyngeal swallowing.

Functional Oral Intake Scale

Intervention Type: OTHER

The Functional Oral Intake Scale (FOIS) will be used for participants to report their food intake habits.

Eating Assessment Tool-10

Intervention Type: OTHER

The Eating Assessment Tool-10 will be used for participant reporting of swallowing system severity.

Interventions

Videofluoroscopic swallowing study

Videofluoroscopic swallowing study will be performed to measure the oropharyngeal swallowing.

Intervention Type: PROCEDURE

Functional Oral Intake Scale

The Functional Oral Intake Scale (FOIS) will be used for participants to report their food intake habits.

Intervention Type: OTHER

Eating Assessment Tool-10

The Eating Assessment Tool-10 will be used for participant reporting of swallowing system severity.

Intervention Type: OTHER

Other Intervention Names

VFSS; modified barium swallow (MBS); FOIS; EAT-10

Eligibility Criteria

Inclusion Criteria

• diagnosis of oculopharyngeal muscular dystrophy
• no allergies to barium or capsaicin,
• no tracheotomy or mechanical ventilation
• no significant concurrent respiratory disease (e.g., COPD).

Exclusion Criteria

• Pregnant Women

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Florida

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Emily Plowman, PhD

Role: PRINCIPAL_INVESTIGATOR

University of Florida

Locations

University of Florida

Gainesville, Florida, United States

Countries

United States

Other Identifiers

IRB201601374

Identifier Type: -

Identifier Source: org_study_id